Artificial mini-brains for AI powered drug screening to improve success rates of clinical trials in brain disorders

Purpose and goal:

More than a billion people suffer from neurological diseases like depression, schizophrenia or Alzheimer’s disease; new therapies are hard to develop due to limited understanding of human brain physiology. In neuroscience, only 6% of new medicine candidates succeed in clinical trials.

We aimed to build and validate a technological platform based on drug screening data from human brain organoids minibrains that would allow faster pre-clinical medicine discovery in neuroscience and provide high predictive value of the medicine efficacy and toxicity in patients. Expected results and effects:

We developed a large production and testing platform of human minibrains (cortical organoids) as a more relevant drug discovery tool that would predict outcomes better than animal tests. We screened 20 000 minibrains with 58 neurological medicines and verified that our platform can detect similar symptoms as in humans.

This opens new possibilities to identify new neurological compounds faster, improve the success rate of clinical trials and transform the pharmaceutical industry. Approach and implementation:

From a set of smaller pilot studies that helped us develop robust and reliable screening assays, we moved to large drug screening study that employed robotic handling and automated high-throughput microscopy. Unique expertise of our team in industrial drug screening, method troubleshooting and brain tissue culture brought success, supported by robust data analysis.

Robustness and reliability of the results were highly appreciated in customer feedback during our commercialization efforts, in which we will continue after the project ends.